One way to improve clinical trial reporting: a Yelp-style rating system

This piece originally appeared in the Timmerman Report.

STAT recently published an in-depth report about the many research centers that don’t bother to publicly disclose the results of their clinical trials, even though they are required to do so. This follows on a New England Journal of Medicine article back in March that had a similar analysis of the lack of reporting and publication of clinical trial data to clinicaltrials.gov.

Most observers of biomedical research would agree that getting clinical trial data out about what happened in a trial is pretty important, whether the trial succeeded or failed. After all, biomedical translational research is most meaningful when done on human subjects and negative information can be quite informative and useful. Animal models are nice, but translation of results from animals to humans is a spotty proposition at best. We need to know what’s working, and what’s not, to know how to best allocate our research resources and how to treat patients.

The lack of reporting is an embarrassment for research. It’s also understandable, because so far the FDA hasn’t used its authority to punish anyone for delayed reporting. Nobody appears to have lost any research funding because they failed to post trial results in a timely manner. Universities told STAT their researchers were “too busy,” given other constraints on their time, to report their results. So what really seems to be going on is that reporting is prioritized below most other activities in clinical research.

It was interesting and eye-opening that industry fared better than academia in both the STAT story and the NEJM article with respect to how many studies have been reported. Having seen the industry process first-hand, I’d speculate that (at least for positive trials) there’s a much stronger incentive to get data out in public. Successful trial results can create buzz among clinicians and patients, revving up trial enrollment which can then help get a new drug on the market faster, and convince people to use it when it’s available. It may be that in academia the effort of getting trial results in the required format for clinicaltrials.gov is perceived as too much work, relative to the rewards. Academics are naturally going to spend more energy on directly rewarded activities like writing grant proposals and writing peer-reviewed scientific publications that help them win even more grants, promotions, and other accolades. Well okay. If this is the case, then figuring out new incentives may be key.

So what would work? Anyone who participates in a clinical trial is providing time, may be subject to risks and often is asked to provide samples that are biobanked to support future exploratory and translational research. It’s like when people donate to food banks. I’m pretty sure they mean that food to be eaten and not to sit on a shelf. These participants in clinical trials deserve to have their volunteerism rewarded.

This got me thinking about how to empower patients to get more of what they want. Patient-centered research is a buzzword these days, and for good reason. Patients have at times been an afterthought in the biomedical research enterprise. I thought of services like Yelp and Uber and Angie’s List and other peer-to-peer systems that allow users to get information, provide feedback and give ratings to specific providers. And I wondered: could this be a way to apply pressure to clinical trial researchers to improve their reporting? Continue reading

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The innovators dilemma in biopharma part 3. What would disruption look like?

All opinions are my own and do not necessarily reflect those of Novo Nordisk.

h/t to @Frank_S_David, @scientre, and the LinkedIn Group Big Ideas in Pharma Innovation and R&D Productivity for links and ideas

Part 1 is here.

Part 2 is here.

In the previous parts to this series I’ve covered both why the biopharma industry is ripe for disruption, and what the markets might be that could support a nascent, potentially disruptive technology until it matures enough to allow it to supplant the current dominant industry players.  In this final part I’d like to ask what disruption would look like and provide some examples of directions and companies that exemplify what are, to my mind, these sorts of disruptive technologies and approaches. With, I might add, the complete and utter knowledge that I’m wrong about who and what specifically will be disruptive! But in any case, before we can identify disruption, it’s worthwhile to ask what are the key elements of biopharma drug development that serve as real bottlenecks to affecting  human health, since these are the elements most likely to provide an avenue for disruption. Continue reading

The Innovator’s Dilemma in biopharma part 2. Where are the markets for disruptive tech?

All opinions are my own and do not necessarily reflect those of Novo Nordisk.

h/t to @Frank_S_David, @scientre, and the LinkedIn Group Big Ideas in Pharma Innovation and R&D Productivity for links and ideas

Biopharma may be ripe for disruptive innovation to come in and overturn their markets but that doesn’t mean it will happen. There are constraints beyond those of pure business, including the simple fact that treating diseases is really difficult and we don’t know as much as we would like about how biology really works. I see today’s biopharma market as a victim of its own success. The 80s and 90s saw the creation of truly life-changing, effective drugs like statins, and that has set the bar high enough that I think we’ve passed the inflection point at which approaches like high-throughput screening are becoming less likely to yield a substantial improvement in effectiveness. I’ve used the analogy before of drug development occurring on an adaptive landscape (Figure 2), with every improvement moving up a hill towards the theoretical perfect medicine at the apex. The higher up the hill one gets, the harder it is to move uphill and most efforts move sideways or down, simply because there’s more territory in those directions. This is a constraint that a disruptive innovation would have to overcome in some way.

Figure 2

Figure 2: The adaptive landscape for drug development.  Yes I drew this myself.  I would plug the drawing program, except I think they’d probably prefer not to be associated with this image. Continue reading

The Innovator’s Dilemma in biopharma part 1. Framing the industry’s position

All opinions are my own and do not necessarily reflect those of Novo Nordisk

h/t to @Frank_S_David, @scientre, and the LinkedIn Group Big Ideas in Pharma Innovation and R&D Productivity for links and ideas

Joe Nocera’s recent column in the New York Times provided a nice dissection of how Blackberry tumbled from the position it once held at the top of the handheld phone/PDA business market.  In a nutshell it encapsulates how Blackberry fell victim to the Innovator’s Dilemma, the paradigm put forward by Clay Christensen about how and why established companies within an industry often fall victim to disruptive technologies.  This happened even though they were aware of the danger and made efforts to circumvent the dilemma.  In the case of Blackberry, one aspect of their fall was a lack of appreciation for the technology creeping up behind: the iPhone and other mobile devices using touchscreens.  For Blackberry one of their advantages and selling points was a physical keyboard which allowed rapid typing and emailing by business customers.  They couldn’t see why anyone would want something less effective for emails and messaging.

In addition, Blackberry felt both secure in and beholden to their customer base, the businesspeople who used Blackberries strictly as tools for work.  Blackberry (Research in Motion at the time) seemed both unable to conceive of the possibility of other markets and, frankly, had no incentive to reach into those markets until it was too late.  By then other phones and operating systems had grown and matured to the point of essentially overtaking the market of smartphone users, of which businesspeople make up just a small fraction.  Too little, too late, and now Blackberry has been trying to sell itself, although recent reports suggest that strategy is also failing.

From Blackberry to biopharma

In this post I’d like to explore the concept of the Innovator’s Dilemma as it might apply to the biopharmaceuticals industry.  Continue reading